Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia.
Suzanne CrowleyInês AzevedoMieke BoonAndrew BushErnst EberEric HaarmanBulent KaradagKarsten KötzMargaret LeighAntonio Moreno-GaldóHuda MussaffiKim Gjerum NielsenHeymut OmranJean-François PaponPetr PohunekKostas PriftisBernhard RindlisbacherFrancesca SantamariaArunas ValiulisMichal WittPanayiotis YiallourosZorica ZivkovicClaudia Elisabeth KuehniJane S A LucasPublished in: ERJ open research (2020)
Primary ciliary dyskinesia, a rare disease causing bronchiectasis, lacks a sound evidence base for treatment. @beatpcd proposes 1) forming a PCD European clinical trial network to address this situation and 2) conducting n-of-1 trials to access medication. https://bit.ly/3j5blfM.